Brineura

USDailyMed:Cerliponase_alfa A16AB17(WHO) CA:℞-only/ Schedule DUS:℞-onlyEU:Rx-only 151662-36-1 319902614 DB13173 X8R2D92QP1 D10813 ChEMBL3544921 Cerliponase alfa, marketed asBrineura, is an enzyme replacement treatment forBatten disease, a neurodegenerative lysosomal storage disease.​​​‍​​‍​​​​‍​​‍‍ Specifically, Cerliponase alfa is meant to slow loss of motor function in symptomatic children over three years old withlate infantile neuronal ceroid lipofuscinosis type 2(CLN2). The disease is also known astripeptidyl peptidase-1 (TPP1)deficiency, a soluble lysosomal enzyme deficiency.Approved by the United StatesFood and Drug Administration(FDA) on 27 April 2017, this is the first treatment for aneuronal ceroid lipofuscinosisof its kind, acting to slow disease progression rather than palliatively treat symptoms by giving patients the TPP1 enzyme they are lacking. The U.S.Food and Drug Administration(FDA) considers it to be afirst-in-class medication.